Developing target product profiles for innovation in cancer diagnosis

What is the issue?

Detecting and diagnosing cancer early can help improve patient prognosis and is a key goal of efforts to tackle cancer internationally. In the United Kingdom, policymakers have set out an ambition to increase the proportion of cancers diagnosed early from approximately 50% to 75% by 2028 (NHS Long Term Plan, 2019). Such efforts can be supported by the development and adoption of improved diagnostic tests.

Those who might use and pay for diagnostic tests need to provide a clear demand signal to innovators on types of tests that are needed, so that innovators can appropriately respond to areas of unmet need. Diagnostic Target Product Profiles (TPPs) are a tool that help in this regard and in doing so help in efforts to align supply of much needed innovation with demand. Diagnostic TPPs are product specification documents. They can support the development and assessment of new diagnostic technologies/products by providing clear specifications on the types of features a novel test needs to meet. They can include information about the technology/product itself and about the context in which the technology/product will be used, in order to help ensure a good fit.

Diagnostic TPPs have been developed for other areas, such as infectious diseases, but their potential for use in oncology has been underexplored.

How did we help?

Cancer Research UK commissioned RAND Europe, working in collaboration with the Office of Health Economics (OHE) and Professor Larry Kessler from the University of Washington, to conduct a project to advance practical knowledge on approaches to developing diagnostic TPPs for cancer and on desired TPP features. The overarching goal is to support policy and practical efforts to enable earlier cancer detection and diagnosis by helping align supply with demand, ultimately benefitting patients.

This project established a ‘general’ (tumour-site agnostic, i.e. for cancer generally regardless of where it started in the body) guide for developing oncology TPPs. This is an evidence-based tool that provides information on the process by which a diagnostic TPP for cancer can be developed and the types of features that need to be considered in a TPP. The guide serves as a practical resource for future efforts to develop bespoke TPPs for specific cancers, test types and use cases.

The project adopted a mixed-methods approach involving scoping desk research and interviews, workshops with diverse stakeholder communities, and the establishment of an early economic modelling tool to help with efforts to specify requirements in a TPP.

What did we find?

• TPPs need to provide specifications for a diverse range of features that reflect key requirements for successful real-world use of a diagnostic test. There are different types of features that can be relevant (e.g. as related to unmet need, scientific and technical performance of a test, interactions between the test and a human user, infrastructure, regulation, economic and environmental considerations).
• Developing TPPs takes time and effort. It is important to avoid the risks of trying to achieve too much at the expense of prioritising what matters most. Establishing specifications for combinations of core features driving a test’s value is key to TPP development. Trade-offs between different requirements must be considered. Early economic modelling can help.
• Given the diversity of factors that influence the extent to which an innovative test is fit for use in practice, developing TPPs requires input from diverse stakeholders. This includes academics/researchers; healthcare professionals and diagnostic laboratory expertise; industry; patient, carer and public perspectives; research and innovation funders; and wider regulator, health technology assessment, policymaker/payer perspectives.
• TPP development has two key stages: inception (establishing core working group, governance, action plan) and implementation (scoping unmet need and key test requirements, TPP drafting, consensus building).
• A range of methods can be used to develop TPPs, including different types of literature reviews and desk research, stakeholder consultation and consensus exploration, and economic modelling. It is important to consider both rigour and feasibility when selecting which methods to use. This can be influenced by various factors such as the urgency of need for a new test, resources available, and the strength of the pre-existing evidence base.

Recommendations

• Robust TPPs are an important tool for developing improved cancer diagnostic tests, but efforts to develop TPPs as demand signalling tools need to align with wider innovation and health system efforts.
• Given the needs for diagnostic innovation for many different types of cancers, it will be important to prioritise which use cases to develop TPPs for. This needs to consider both clinical needs, as well as viability of demand and willingness to pay.
• Key decision-makers in a health system need to be engaged in early discussions to help prioritise which TPPs should be developed. This is because policy and NHS priorities in the cancer diagnosis space and funding programmes for purchasing tests will impact on the likelihood of a test that is developed in response to a TPP being adopted in the health system.
• Many of the insights gained through this work may be applicable to other clinical areas and contexts. A future research agenda could further explore how the guide we have developed can be applied and adapted to diverse use cases.